Clinical Trials 101

The following are a series of Frequently Asked Questions (FAQ) pertaining to clinical trials, and what the process entails:

What is a clinical trial?

Clinical trials are well-designed studies that collect information about new treatments for diseases and disorders. Most of the time, this means medications, but clinical trials can also test other things, such as stem cell therapies, surgical techniques, tests for diagnosis, medical devices, as well as others. In particular, clinical trials focus on administering an experimental therapy in humans, as opposed to animals, which are conducted first in the lab (known as “pre-clinical research”).

Why do we need clinical trials?

Clinical trials are needed for medical treatments to be approved by government organizations, such as the US Food and Drug Administration (FDA). Without clinical trials, doctors and other prescribing healthcare providers (such as nurse practitioners or physician’s assistants) cannot prescribe medications or recommend other medical treatments. These studies are needed to understand two important types of information 1) that the treatment is effective (also called efficacious), ie, that it really works and 2) that the treatment is safe for use in humans.

In addition to helping patients by making the best possible treatments available, clinical trials also advance scientific understanding of a disease or disorder.

How do clinical trials work?

Usually, effectiveness is compared versus a placebo (sugar pill with no medication in it), or another type of comparison. Another type of medication comparison that is common is a different drug, already approved for use. A comparison is needed to understand that the medication works and also to see if the medication is safe. Researchers will design the clinical trial for a specific period of time, during which the people participating will either get the treatment, or the comparison treatment. Typically a study is done “double-blind,” which means that neither the researchers giving the treatments nor the participants know who is getting which treatment. This prevents “bias,” which means expectations that could influence the outcome of the study.

Researchers giving the treatment will have a code that is later “unblinded” so they find out what treatment they were giving. The researchers also record measurements while the participants are receiving the treatment. These measurements can be for different things, such as to determine if the treatment is working, to assess safety and side-effects. Other measurements might include blood levels of the medications. If someone participates in a clinical trial, they will be informed about the measurements that will be taken before the trial starts. An “Informed Consent” document tells participants about the trial.

What kind of clinical trials are there?

The type of trial that most people read about and consider enrolling in is called an interventional trial. This tests treatments that are not yet approved for use. Prevention trials are designed to stop medical conditions from occurring. Observational trials are used to look at health issues in large groups of people, and do not involve treatment. Diagnostic and screening trials are intended to detect and diagnose medical problems.

What are the phases of clinical trials?

Developing a new medical treatment takes many years. The process is intended to best treat medical conditions without harming people. Before a medical treatment can even be tested in people, animal testing is absolutely required. As mentioned above, this phase is called pre-clinical testing. Animal testing is governed by three principals: 1) to reduce the use of animals to the minimum but still collect data indicating that the treatment is safe and effective in people, 2) minimize animal suffering and assure animal welfare as much as possible, 3) replace animal experiments with other alternatives when possible.

Phase I testing is the first step in humans. The purpose is to determine safety and to evaluate side effects. Phase I studies also test how the drug is absorbed, distributed and eliminated from the body. Often people who do not have the disease (healthy individuals) participate in Phase I. The number of people involved at this stage is usually small.

Phase II trials are sometimes divided into Phase IIA and Phase IIB. Sometimes these two sub-phases are combined. Phase II trials further assess dosing and are designed to determine the best drug dose to use and how much of a dose is safe. Phase II studies can also measure efficacy and safety testing in small numbers of participants. Often a treatment must pass Phase II in order to proceed to Phase III.

Most reports of medical treatment studies focus on Phase III trials. These are the large trials that are required for a drug or other treatment to receive approval for use. The purpose of this phase is to test efficacy and safety as well as to monitor for side effects. The main drug effects are often called the primary efficacy endpoints. Other measurements may be called the secondary endpoints.

Sometimes researchers conduct Phase IV trials, after a drug has been approved. These trials collect additional information about the drug or treatment.

Why participate in a clinical trial?

There are several reasons to participate in a clinical trial. One is to get access to a treatment that is not otherwise available. The treatment may be better than others available for the condition. A risk however, could be that the treatment is not better or even has side effects. Treatments in clinical trials are typically free to the participants.

Clinical trials also help to advance science and to generally help other people with the medical condition being studied. They may help with making a new treatment available, but could also improve the general understanding of the disease.

Sometimes there are other incentives to participating in a clinical trial. Participants may receive some reimbursement or they may receive additional benefits, such as counseling or educational materials. These benefits are detailed in any documentation that the participants are given before the trial starts.

How is privacy protected in a clinical trial?

People who participate in a clinical trial are anonymous except to the professionals conducting the trial. These professionals are not allowed to reveal the identity of people participating in the trial to anyone who is not involved in the study.

Personal information that is collected during the trial has to be protected, for example, in locked file drawers or in password protected computers.

Any reports that are made available to the public about the data collected in a clinical trial cannot have any information that identifies the people participating.

Can participants leave a clinical trial once it has started?

It is the right of the participant to leave a clinical trial for any reason at any time. If a participant is receiving a treatment, it is important to consult with a physician to make sure that it is safe to stop the treatment or to help taper off the treatment or replace it with a different treatment.

What happens when the trial ends?

After the trial ends, the investigators will begin to analyze the data that has been collected during the trial. They will write up reports about what they have found and submit them to a treatment approving agency, such as the FDA. The agency will make decisions about whether the treatment will move further into other trials, or whether it will be approved.

The study researchers may also write reports about what they have found in the trial that are made available to the public. These reports could be published as presentations at medical conferences, or as scientific papers in medical journals. Often a drug company will issue press releases on their web site, describing where the data has been presented.

Do participants still get treatment after the trial ends?

After the trial ends, treatment may end, or participants may be offered continued treatment. This is frequently referred to as an “open label extension.” Participants who have received placebo are sometimes able to get the treatment at the end of a study as well. To understand what happens after the trial ends, it is important to speak with the individuals conducting the trial to see if the option exists to continue the treatment after the trial has ended. Participants should also review any documentation describing the trial, such as the Informed Consent documentation. A doctor could also make a decision to continue the participant on the treatment (if it is available) or a similar treatment.

Can someone get a medication that has not gone through a clinical trial?

All medications must go through clinical trials to be used in patients. It is possible for physicians and other prescribing healthcare providers to prescribe a medication that has gone through clinical trials for a different use. This is called “off-label use.” It means that the treatment went through a clinical trial, usually for a similar condition. Using a medication off-label is based on the clinical judgement of the person prescribing the medication.

If a drug works, why can’t we just start giving it to people right away?

It is important to make sure that a drug is more effective than existing treatments, actually works in a disease and that it is safe. Decisions to give drugs or other medical treatments have to come from scientific data, not just the opinion of a healthcare provider, patient or other individual. Clinical trials help to assure all of these things.

Isn’t there a way to get treatments for serious medical conditions approved for use faster?

The FDA does have what is called a Fast Track Designation, which helps to make drugs available faster if they are used for a life-threatening condition or if they can help fulfill an unmet medical need. A drug company developing the treatment needs to request Fast Track Designation. This can be done at any time during the treatment development process. After a drug company requests Fast Track Designation, the FDA has 60 days to make a decision.

How can someone participate in a clinical trial?

Companies developing treatments often have contact information available on their websites that interested participants use to find out how to be included in a trial. The medical centers conducting the trials may also have contact information. Trials conducted in the United States are registered with Clinicaltrials.gov, and individuals responsible for enrolling trials will have their contact information listed there. CenterWatch is another site that helps connect people with clinical trials.

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Disclaimer:

IBD News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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